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Sanofi-GSK next-generation COVID-19 booster delivers strong immune response

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Sanofi-GSK next-generation COVID-19 booster delivers strong immune response

June 13, 2022: “Sanofi reports data from two trials, VAT02 Cohort 2 and COVIBOOST VAT013, conducted with its new next-generation COVID-19 booster vaccine candidate modelled on the Beta variant antigen and including GSK’s pandemic adjuvant.

In the Phase 3 VAT02 Cohort 2 study, the Sanofi-GSK next-generation vaccine candidate induced (at day 15 post-immunization) a significant boost in antibody titers above baseline against multiple variants of concern (15-fold increase against D614 parent virus, 30-fold increase against Beta strain) in adults previously primed with mRNA COVID-19 vaccines.

In particular against Omicron, preliminary data show a 40-fold increase against BA.1.

The Sanofi-GSK next-generation booster candidate generated double the number of neutralizing antibodies against Omicron BA.1 and BA.2 compared to the D614-based (original parent virus) booster.

In parallel, the independent COVIBOOST (VAT013) study conducted by the Assistance Publique – Hôpitaux de Paris (AP-HP) demonstrated that, following primary vaccination with two doses of Pfizer-BioNTech’s Comirnaty vaccine, the Sanofi-GSK next-generation booster candidate generated a higher immune response (as measured by neutralizing antibody titers) than Pfizer-BioNTech’s booster or the Sanofi-GSK first-generation booster, both of which target the original D614 parent strain.

The proportion of participants with at least a 10-fold increase in neutralizing antibody titers for the original D614 SARS-CoV-2 strain between day 0 and day 15 was:

  • 76.1% (95% CI 64.5–85.4) for the Sanofi-GSK next-generation booster, vs
  • 63.2% (95% CI 51.3–73.9) for the Pfizer BioNTech D614 booster, and
  • 55.3% (95% CI 43.4–66.7) for the Sanofi-GSK D614 (first-generation parent booster candidate).

In this study, which included 247 subjects, all the three vaccines also elicited neutralizing antibodies against the Omicron BA.1 variant, with highest responses generated by the Sanofi-GSK next-generation candidate.

Results of COVIBOOST study are available on a pre-print server, pending publication in a peer-reviewed journal.Across both studies, the Sanofi-GSK next-generation vaccine candidate was well-tolerated, with a favorable safety profile. In the VAT02 cohort 2 study, low numbers (less than 4%) of Grade 3 reactions were reported, all transient and non-severe.

Thomas Triomphe
Executive Vice President, Sanofi Vaccines
“COVID-19 keeps evolving and the combination of emergence of variants and waning immunity is likely to lead to the need for additional booster shots, at least in some populations. 

The Beta variant expresses similar mutations across multiple variants of concern, including Omicron, making it a strong vaccine candidate to confer broad protection against multiple strains of COVID-19. 

Seeing the cross-neutralization data from the independent AP-HP study, we believe this next-generation booster could have an important role to play for public health vaccination campaigns.

We look forward to submitting these data to global regulatory authorities.”

Sanofi and GSK have developed their next-generation booster candidate in parallel to ongoing regulatory reviews of their first-generation vaccine candidate.

The totality of data supporting this next-generation booster vaccine will be submitted to regulatory authorities in the upcoming weeks, with the aim of making it available later this year.

About VAT02
The VAT02 booster study is an extension of the company’s phase 3 safety and immunogenicity study.

In Cohort 1 of this study, participants previously vaccinated with the primary series of an authorized COVID-19 vaccine received a booster dose of the Sanofi-GSK adjuvanted recombinant vaccine candidate, using SARS-CoV-2 (D614) antigen.

These data confirmed the vaccine candidate’s universal potential to boost neutralizing antibodies 18- to 30-fold across all vaccine platforms (mRNA, protein, adenovirus). Cohort 2 included 1,500 participants.

VAT02 results will be published in a peer-reviewed journal at a later date.

These efforts are supported by federal funds from the Biomedical Advanced Research and Development Authority, part of the office of the Assistant Secretary for Preparedness and Response at the U.S.

Department of Health and Human Services in collaboration with the U.S. Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense under Contract # W15QKN-16-9-1002 and by the National Institute of Allergy and Infectious Diseases (NIAID).

The NIAID provides grant funding to the HIV Vaccine Trials Network (HVTN) Leadership and Operations Center (UM1 AI 68614HVTN), the Statistics and Data Management Center (UM1 AI 68635), the HVTN Laboratory Center (UM1 AI 68618), the HIV Prevention Trials Network Leadership and Operations Center (UM1 AI 68619), the AIDS Clinical Trials Group Leadership and Operations Center (UM1 AI 68636), and the Infectious Diseases Clinical Research Consortium (UM1 AI 148684, UM1 AI 148450, UM1 AI 148372 , UM1 AI 148574).

About COVIBOOST (VAT013) study
COVIBOOST is an independent study conducted by the Assistance Publique – Hôpitaux de Paris (AP-HP).

It is a randomized, single-blinded, multicenter trial across 11 centers in France, which studies the immune response of the Sanofi-GSK first- and next-generation booster vaccine candidates (adjuvanted, recombinant protein) and that of a 3rd dose of the Pfizer-BioNTech vaccine Comirnaty, following two doses of Comirnaty received as primary vaccination.

The study was funded by the French Ministry of Solidarity and Health and Sanofi.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-13-05-30-00-2460833

Novartis five-year Kymriah® data show durable remission and long-term survival

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Novartis five-year Kymriah® data show durable remission and long-term survival

June 12, 2022: “Novartis announced long-term results from the ELIANA pivotal clinical trial of Kymriah® (tisagenlecleucel), the first-ever approved CAR-T cell therapy, in children and young adult patients with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (ALL), with a maximum survival follow-up of 5.9 years.

For the 79 patients treated with Kymriah in this study, the five-year overall survival (OS) rate was 55% (95% CI, 43-66), while the median event-free survival (EFS) for patients in remission within three months of infusion (n=65) was 43.8 months.

These findings demonstrate the curative potential of Kymriah, the only CAR-T cell therapy available for these patients who previously had limited treatment options.

These data were presented as an oral presentation during the 2022 European Hematology Association (EHA) Hybrid Congress (Abstract #S112)1.

“These data mark a moment of profound hope for children, young adults and their families with relapsed or refractory B-cell ALL, as relapse after five years is rare,” said Stephan Grupp, MD, PhD, Section Chief of the Cellular Therapy and Transplant Section, and Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at Children’s Hospital of Philadelphia (CHOP).

“Since the approval of Kymriah nearly five years ago, we have been able to offer a truly game-changing option to patients who previously faced a five-year survival rate of less than 10 percent.”

This long-term follow up of ELIANA demonstrated the potential for Kymriah to transform cancer treatment in pediatric and young adult patients with r/r B-cell ALL, significantly improving outcomes with durable responses and a consistent safety profile in this patient population:

  • Eighty-two percent of patients experienced remission (either complete remission [CR] or CR with incomplete hematologic recovery within three months after infusion) (95% CI, 72-90)
  • For patients in remission, the five-year relapse-free survival (RFS) rate was 44% (95% CI, 31-56) and the median RFS was 43 months
  • No new or unexpected adverse events were reported during long-term follow-up

“At Novartis, we strive for cures. With nearly six-year follow-up data in these pediatric and young adults treated for B-cell ALL, we have our strongest evidence yet that one-time treatment with Kymriah has curative potential,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development.

“These results strengthen our confidence in CAR-T cell therapies as a truly transformative and paradigm-shifting advance in cancer care, as well as our commitment to continue developing this technology with next-generation platforms.”

Additional updates on the Novartis CAR-T program presented at the 2022 EHA Congress include new data from more patients and longer follow-up from the first-in-human dose-escalation trials with YTB323 in adults with r/r diffuse large B-cell lymphoma and PHE885 in adults with r/r multiple myeloma, the first Novartis CAR-T cell therapies developed using the Novartis T-Charge™ platform.

Visit https://www.hcp.novartis.com/virtual-congress/eha-2022/ to learn more about these data and our ongoing commitment to reimagining cancer care with CAR-T cell therapies.

About Kymriah®
Kymriah is the first-ever FDA-approved CAR-T cell therapy.

It is a one-time treatment designed to empower patients’ immune systems to fight their cancer.

Kymriah is currently approved for the treatment of r/r pediatric and young adult (up to and including 25 years of age) acute lymphoblastic leukemia (ALL), r/r adult diffuse large B-cell lymphoma (DLBCL) and r/r adult follicular lymphoma.”

https://www.novartis.com/news/media-releases/novartis-five-year-kymriah-data-show-durable-remission-and-long-term-survival-maintained-children-and-young-adults-advanced-b-cell-all

AZ demonstrates pipeline and portfolio strength across malignant and rare haematological diseases at EHA 2022

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AZ demonstrates pipeline and portfolio strength across malignant and rare haematological diseases at EHA 2022

June 09, 2022: “AstraZeneca will showcase data demonstrating the Company’s commitment to redefine care in haematology at the European Hematology Association (EHA) Annual Meeting, 9 to 12 June 2022.

A total of five approved and potential new medicines from AstraZeneca will be featured across 16 abstracts, including one oral presentation.

These include long-term follow-up data for Calquence (acalabrutinib) in adults with previously untreated and relapsed or refractory chronic lymphocytic leukaemia (CLL).

Additionally, Alexion, AstraZeneca’s Rare Disease group, will present an analysis of long-term survival data from the clinical trial programme evaluating Ultomiris (ravulizumab) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).

One-year safety and efficacy data from the Phase III clinical trial evaluating the subcutaneous administration of Ultomiris will also be presented.

Anas Younes, Senior Vice President, Haematology R&D, AstraZeneca, said: “At this year’s European Hematology Association Annual Meeting, we are demonstrating our strength across a broad spectrum of haematological malignancies, and specifically in chronic lymphocytic leukaemia, to help improve outcomes for these patients.

The data from our portfolio and pipeline represent the outcome of years of dedication and passion focused on delivering improved treatment options that can have a long-term impact for patients with chronic, hard-to-treat and rare blood conditions.”

Christophe Hotermans, MD, PhD, Senior Vice President, Global Medical Affairs, Alexion, said: “The collective clinical and real-world data being presented at the European Hematology Association Annual Meeting will strengthen the body of scientific evidence supporting the effective and well-tolerated use of targeted C5 complement inhibition, which is the standard of care in paroxysmal nocturnal haemoglobinuria.

The data reinforce targeting of the terminal complement system through C5 inhibition to reduce intravascular haemolysis and thrombosis in this devastating disease.”

Redefining expectations for patients with CLL

  • Updated data from the ELEVATE-TN Phase III trial at approximately five years of median follow-up will highlight longer-term safety, progression-free survival (PFS) efficacy results and overall survival rates for Calquence in combination with obinutuzumab and alone compared to obinutuzumab plus chlorambucil in adults with previously untreated CLL.
  • Updated data from the ASCEND Phase III trial at approximately four years of median follow-up will highlight longer-term safety and PFS efficacy results of Calquence alone compared to investigator’s choice of idelalisib plus rituximab or bendamustine plus rituximab in adults with relapsed or refractory CLL.
  • A pooled analysis of data among previously untreated and relapsed or refractory CLL patients with higher-risk genomic features taking Calquence will explore the efficacy of Calquence-based regimens for these sub-groups of patients, regardless of line of therapy.
  • A crossover analysis from the ELEVATE-TN trial will test the hypothesis that treatment crossover upon disease progression from the obinutuzumab plus chlorambucil arm to the Calquence arm bolstered overall survival for the obinutuzumab plus chlorambucil arm at 47 months of follow-up.

Improving understanding of hard-to-treat blood cancers

  • A post-hoc analysis of the ACE-LY-004 Phase II trial will highlight the clinical benefit seen with patients taking Calquence who had one prior therapy and with highly proliferative variants of relapsed or refractory mantle cell lymphoma (MCL).
  • Final results at five-year median follow-up from the WM-001 Phase II trial will highlight response rates and tolerability seen with Calquence in patients with previously untreated or relapsed or refractory Waldenström macroglobulinemia (WM).
  • Early results from a Phase II trial will show the efficacy and tolerability of Calquence in patients with relapsed or refractory marginal zone lymphoma (MZL).

Advancing treatment and care for patients with PNH

  • Pooled, long-term, survival data for more than four years from the Ultomiris PNH clinical trial programme will highlight long-term use of Ultomiris in the treatment of adults with PNH.

    Ultomiris has the largest clinical trial programme in PNH, with the longest follow-up, and demonstrates the benefits of immediate, complete and sustained terminal complement inhibition in this rare disease.
  • Data through one year on the efficacy, safety and treatment administration satisfaction will be presented from the Phase III clinical trial evaluating the subcutaneous administration of Ultomiris with an on-body delivery system in adults with PNH who had received prior intravenous treatment with Soliris (eculizumab).

Key AstraZeneca presentations during EHA 2022

Lead authorAbstract titlePresentation details
Calquence (acalabrutinib)
Sharman, JPAcalabrutinib ± Obinutuzumab vs Obinutuzumab + Chlorambucil in Treatment-Naive Chronic Lymphocytic Leukemia: 5-Year Follow-up of ELEVATE-TN Abstract # P666Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Ghia, PAcalabrutinib vs Rituximab Plus Idelalisib or Bendamustine in Relapsed/Refractory Chronic Lymphocytic Leukemia: ASCEND Results at ~4 Years of Follow-up Abstract # P668Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Davids, MSLong-term Efficacy of Acalabrutinib-Based Regimens in Patients With Chronic Lymphocytic Leukemia and Higher-risk Genomic Features: Pooled Analysis of Clinical Trial Data Abstract # P667Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Gaitonde, PAdjusting Survival Data for Treatment Crossover in the ELEVATE-TN Trial by Using a Historical Cohort of Patients Treated with Chemoimmunotherapy in Front-Line Chronic Lymphocytic Leukemia Abstract # PB1877e-PublicationOnline Only12 May 2022
Le Gouill, SPost Hoc Analysis of Patients With Highly Proliferative Variants of Mantle Cell Lymphoma Treated With Acalabrutinib Abstract # P1131Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Owen, RAcalabrutinib in Treatment-Naive or Relapsed/Refractory Waldenström Macroglobulinemia: 5-Year Follow-up of a Phase 2, Single-Arm Study Abstract # P1130Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Strati, PAcalabrutinib in Patients With Relapsed/Refractory (R/R) Marginal Zone Lymphoma (MZL): Results of a Phase 2, Multicenter, Open-label Trial Abstract # P1129Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Ultomiris (ravulizumab)
Kulasekararaj, ALong-Term Complement Inhibition and Survival Outcomes in Patients with Paroxysmal Nocturnal Hemoglobinuria: An Interim Analysis of the Ravulizumab Clinical TrialsAbstract # P812Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Yenerel, MEfficacy, Treatment Administration Satisfaction and Safety of Subcutaneous Ravulizumab Through 1 Year in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous EculizumabAbstract # P813Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
Soliris (eculizumab)
Nishimura, JReal-World Outcomes of Eculizumab Treatment in Patients with Paroxysmal Nocturnal Hemoglobinurea: A Systematic Literature Review and Evidence SynthesisAbstract # PB1934e-PublicationOnline Only12 May 2022
Rovó, AReal-World Evidence of Safety and Effectiveness of Eculizumab and Switch to Ravulizumab in a Swiss Patient Population With Paroxysmal Nocturnal HemoglobinuriaAbstract # P834Poster PresentationPoster Session10 June 202216:30 – 17:45 CEST
ALXN1820
Kim, SProperdin-Blocking Antibodies Attenuate Complement Alternative Pathway Activation Triggered by Cell-Free Heme in Sickle Cell Disease ModelsAbstract # S267Oral PresentationSession – Sickle Cell Disease: Novel Biomarkers and Therapies12 June 202211:30 – 12:45 CEST

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/astrazeneca-demonstrates-pipeline-and-portfolio-strength-across-malignant-and-rare-haematological-diseases-at-eha-2022.html

FDA accepts Dupixent® for priority review in adults with prurigo nodularis

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FDA accepts Dupixent® for priority review in adults with prurigo nodularis

May 31, 2022. The U.S.FDA has accepted for priority review the sBLA for Dupixent® (dupilumab) to treat adults with prurigo nodularis, a chronic inflammatory skin disease that causes extreme itch and skin lesions.

The target action date for the FDA decision is September 30, 2022.

The sBLA is supported by data from two pivotal Phase 3 trials evaluating the efficacy and safety of Dupixent in patients 18 years and older with uncontrolled prurigo nodularis (PRIME2 and PRIME).

Both trials met the primary and key secondary endpoints, showing Dupixent significantly improved disease signs and symptoms compared to placebo, including reduction in itch and skin lesions.

The safety results from these trials were generally consistent with the known safety profile of Dupixent in atopic dermatitis. The adverse event more commonly observed with Dupixent was conjunctivitis.

The FDA grants priority review to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions.

Additional regulatory filings outside of the US are also planned in 2022. The potential use of Dupixent in prurigo nodularis is currently under clinical development, and the safety and efficacy have not been fully evaluated by any regulatory authority.

About Prurigo Nodularis
People with prurigo nodularis experience intense, persistent itch, with thick skin lesions (called nodules) that can cover most of the body.

Prurigo nodularis is often described as painful with burning, stinging and tingling of the skin.

The impact of uncontrolled prurigo nodularis on quality of life is one of the highest among inflammatory skin diseases due to the extreme itch and is comparable to other debilitating chronic diseases that can negatively affect mental health, activities of daily living and social interactions.

High-potency topical steroids are commonly prescribed but are associated with safety risks if used long term.
There are approximately 75,000 people in the U.S. who are unable to control their disease with systemic therapy and are most in need of a treatment option.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-05-31-07-00-00-2452848

Sandoz launched initiative to support improved healthcare access, equity and sustainability

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Sandoz launched initiative to support improved healthcare access, equity and sustainability

May 31, 2022: “Sandoz, a global leader in generic and biosimilar medicines announced the launch of a new global initiative called ‘Act4Biosimilars’ to help address health inequity and inequality worldwide.

Act4Biosimilars aims to increase patient access to advanced medicines by facilitating greater approvability, accessibility, acceptability and affordability (the 4 A’s) of biosimilars.

The initiative is supported by a multidisciplinary Steering Committee of patient advocacy leaders, healthcare professionals, biosimilar experts and industry leaders from around the world, with a mission to increase global adoption of biosimilar medicines by at least 30% in 30+ countries by 2030.

A biosimilar is a successor to a biological medicine (also known as “reference medicine”) for which the patent has expired and exclusivity has been lost.

Biosimilars match their respective reference medicine in terms of quality, safety and efficacy. Hence, the biosimilar can be expected to behave in the same way as the reference medicine in all indications and patient populations that the reference medicines are approved in.

Biosimilars are used in the treatment and prevention of many disabling and life-threatening diseases such as cancer, psoriasis, arthritis, and diabetes.

“As a result of their affordability, biosimilars have opened up a new realm of possibility for patients by enabling biological medicines to become more widely available to those in need of these advanced, life-changing treatments,” said Zorana Maravic, CEO, Digestive Cancers Europe. 

“However, until we address existing biosimilar access inequities, far too many patients will continue to miss out on treatment with the best possible medicines.”

Act4Biosimilars.com outlines the 12 goals that have been identified under the 4As by the Steering Committee to achieve the mission.

These include ensuring equitable pricing, involving patients in treatment decisions and streamlining biosimilar development.

As an immediate next step, the Steering Committee is developing an Action Plan to provide the strategies, tools and activities needed to equip and empower stakeholders to realize these 12 goals, across the 30 countries and beyond.

The Action Plan will be supported by Country Indicator Maps to track and measure the change that is being driven by groups, associations and organizations worldwide.

“While countries such as Norway are leading the way in biosimilar adoption and have successfully realized significant healthcare savings through increasing availability of biosimilars, there are other countries still progressing and aspiring to reach their full potential,” said Professor emeritus Tore K Kvien, previous Head of Department of Rheumatology, Diakonhjemmet Hospital for 25 years.

“Misinformation on the safety, efficacy and science of biosimilars continues to cause confusion and impede uptake. With the Act4Biosimilars Action Plan, we will prioritize the steps needed to help better educate, inform and create action across all countries and regions.”

The Steering Committee will co-create the Action Plan with a group of biosimilar experts and key stakeholders, including patient organizations, healthcare professionals, trade associations, think tanks, government bodies and professional societies.

Act4Biosimilars is supported by founding sponsor Sandoz, a pioneer and global leader in biosimilars, who developed and brought the first biosimilar medicine to patients 16 years ago.

“Healthcare systems are facing more pressure than ever before, which can be attributed to the costs associated with a growing, aging population, more people being diagnosed with chronic diseases and, most recently, the pandemic,” said Richard Saynor, Chief Executive Officer, Sandoz.

“Biosimilars are part of the solution to support a more sustainable healthcare system for all and we are proud to be the founding sponsor of Act4Biosimilars, which will drive action to bring these more affordable treatment options to patients who need them.”

https://www.novartis.com/news/media-releases/sandoz-announces-new-global-act4biosimilars-initiative-improve-patient-access-and-increase-adoption-least-30-30-countries-2030

FDA Efforts to Result in Millions of Additional Bottles of Infant Formula to Further Increase U.S. Supply

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FDA Efforts to Result in Millions of Additional Bottles of Infant Formula to Further Increase U.S. Supply

May 27, 2022: “The U.S. Fda is providing an update on additional steps it has taken that will lead to more infant formula on U.S. store shelves in the coming weeks and months.

“We continue to work around the clock with our government partners and industry to ensure there’s adequate infant formula available wherever and whenever parents and caregivers need it,” said FDA Commissioner Robert M. Califf, M.D.

“Steps like the one the agency is taking today means more infant formula will be available to parents and caregivers in the weeks and months ahead. We will not rest until our shelves are replete with safe and nutritious infant formula.”

Under the agency’s recent increased flexibilities, Bubs Australia plans to provide at least 1.25 million cans of several varieties of its infant formula such as stage 1 and 2 cans of Bubs Organic Grass Fed, Bubs Supreme A2 Beta-Casein Protein and Bubs Easy-digest Goat Milk, that will make at least 27.5 million full-size, 8-ounce bottles.

Some of this product is currently in stock for transport and additional product is being produced in the coming weeks and months. The U.S. Department of Health and Human Services is evaluating options for getting the products to the U.S. as quickly as possible.

The company has information about the products and where to find product once in the U.S. on the Aussie Bubs websiteExternal Link Disclaimer.

The FDA notified Bubs Australia that the agency is exercising enforcement discretion for the importation of certain infant formula products following the review of information provided pertaining to nutritional adequacy and safety, including microbiological testing, labeling, and additional information about facility production and inspection history. 

On Tuesday and Thursday, the FDA also announced steps that will lead to tens of millions of additional bottles of infant formula, including specialty infant formula that is in short supply for infants with certain allergies or critical health conditions.

The agency is leveraging a number of flexibilities to bolster the supply of products that serve as the sole source of nutrition for many infants while ensuring the infant formula can be used safely and provides adequate nutrition.

The FDA remains in further discussions with manufacturers and suppliers regarding additional supply to ensure there’s adequate infant formula available wherever and whenever parents and caregivers need it.

The FDA issued guidance on May 24 that outlined a process by which the agency would not object to the importation of certain infant formula products intended for a foreign market or distribution in the U.S. of products manufactured here for export to foreign countries.

This guidance also may provide flexibilities to those who manufacture infant formula products domestically and may be able to increase further the quantity of domestically produced product for the U.S. market.

The agency has posted a webpage that will be updated with information about additional products headed to the U.S.

The agency’s around-the-clock work as part of the all-of-government efforts has already begun to improve supply and availability.

The agency expects that the measures and steps it is taking, and the potential for Abbott Nutrition’s Sturgis, Michigan, facility to safely resume production in the near-term, will mean more and more supply is on the way or on store shelves moving forward. 

The FDA continues to advise against making infant formulas at home or diluting formula.

Caregivers are encouraged to work with their child’s health care provider for recommendations on changing feeding practices, if needed.

HHS has also released a fact sheet with information to help families find infant formula.

The agency also monitors online marketplaces for fraudulent products and works with major online retailers to remove violative and harmful products offered for sale on their sites.

Additionally, since many of these fraudulent products originate overseas, the agency targets and examines these products at ports of entry.

The FDA also monitors and follows up on various external signals such as consumer complaints about potential counterfeit and fraudulent products.

The FDA will continue to dedicate all available resources to help ensure that infant formula products remain available for use in the U.S. and will keep the public informed of progress updates.”

https://www.fda.gov/news-events/press-announcements/fda-efforts-result-millions-additional-bottles-infant-formula-further-increase-us-supply

FDA Flexibilities, Collaboration to Yield Millions of Bottles of Specialized Medical Infant Formula in Coming Months to Increase U.S. Supply

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FDA Flexibilities, Collaboration to Yield Millions of Bottles of Specialized Medical Infant Formula in Coming Months to Increase U.S. Supply

May 26, 2022: “The U.S. FDA is providing an update on steps it has taken that will lead to millions of bottles of specialized medical infant formula to help address immediate needs for infants with certain allergies or critical health conditions.

This type of specialized medical infant formula is currently in short supply in the U.S.

“We have made tremendous progress, including notable steps in just the past week, which will allow us to immediately begin bringing specialty and infant formula products into the U.S. as quickly as possible,” said FDA Commissioner Robert M. Califf, M.D.

“We continue to work closely with our U.S. government partners and domestic and international manufacturers to identify additional formula product that will be available to parents and caregivers in the weeks and months ahead.

It is our goal to ensure that hospitals, specialty pharmacies, and retail store shelves will begin seeing adequate supplies again in the coming weeks.”

About 500,000 additional cans of specialized medical formula manufactured by Danone’s Nutricia business will be headed to the U.S.

These Neocate amino acid-based formula products — some of which are already manufactured for the U.S. market and are made at facilities in Europe — amounts to more than 5 million full-size, 8-ounce bottles. 

The U.S. Department of Health and Human Services and Danone continue to partner to evaluate options for getting the products to the U.S. as quickly as possible.

The specialized medical formula is expected to be distributed through direct ordering, hospitals, health care professionals, pharmacies, healthcare product distributors and Special Supplemental Nutrition Program for Women, Infants, and Children (WIC) local access points to ensure they get to the infants who rely on these products.

The FDA has been working with Danone’s Nutricia business over the past several months to boost production of these products at its facilities and to identify additional supply of other infant formula products moving forward.

The agency is leveraging a number of flexibilities to bolster the supply of products that serve as the sole source of nutrition for many infants while ensuring the infant formula can be used safely and provides adequate nutrition.

The FDA remains in further discussions with manufacturers and suppliers regarding additional supply to ensure there’s adequate infant formula available wherever and whenever parents and caregivers need it.

On Tuesday, the FDA announced that it informed Kendal Nutricare that the agency is exercising enforcement discretion for the importation of certain infant formula under the Kendamil brand.

Under the agency’s recent increased flexibilities regarding importation of certain infant formula products, the company initially estimates that about 2 million cans of infant formula (over 50 million full-size, 8-ounce bottles) are expected to land on U.S. store shelves beginning in June.

Kendal Nutricare also currently has over 40,000 cans in stock for immediate dispatch.

The FDA also announced that it is not objecting to the release of about 300,000 cans of EleCare amino acid-based infant formula previously produced at Abbott Nutrition’s Sturgis, Michigan, facility to individuals needing urgent, life-sustaining supplies of this specialty formula on a case-by-case basis.

These products will undergo enhanced microbiological testing before release.

Last week, the FDA issued guidance that outlined a process by which the FDA would not object to the importation of certain infant formula products intended for a foreign market or distribution in the U.S. of products manufactured here for export to foreign countries.

This guidance also may provide flexibilities to those who manufacture infant formula products domestically and may be able to increase further the quantity of domestically produced product for the U.S. market. 

The FDA has posted a webpage that will be updated with information about additional products headed to the U.S.

The agency’s around-the-clock work as part of the all-of-government efforts has already begun to improve supply and availability.

The agency expects that the measures and steps it is taking, and the potential for Abbott Nutrition’s Sturgis, Michigan, facility to safely resume production in the near-term, will mean more and more supply is on the way or on store shelves moving forward. 

As more formula becomes available to the general population of infants, the agency understands that availability of specialty products — such as amino acid-based specialty formulas and metabolic products — continues to be of concern.

The FDA has already taken steps with Abbott Nutrition to make product available to those with life-threatening conditions on a case-by-case basis and will continue its efforts to make these products even more readily available as the agency works with the company to implement provisions of a consent decree.

In addition, these products have been an area of focus for discussions with other manufacturers that make comparable products.

As a result of the recall and work with the FDA, other manufacturers have increased production of comparable product lines and in some cases expedited the importation of these products where available.

The FDA continues to advise against making infant formulas at home or diluting formula. Caregivers are encouraged to work with their child’s health care provider for recommendations on changing feeding practices, if needed.

HHS has also released a fact sheet with information to help families find infant formula.

The agency also monitors online marketplaces for fraudulent products and works with major online retailers to remove violative and harmful products offered for sale on their sites.

Additionally, since many of these fraudulent products originate overseas, the agency targets and examines these products at ports of entry.

The FDA also monitors and follows up on various external signals such as consumer complaints about potential counterfeit and fraudulent products.

The FDA will continue to dedicate all available resources to help ensure that infant formula products remain available for use in the U.S. and will keep the public informed of progress updates.”

https://www.fda.gov/news-events/press-announcements/fda-flexibilities-collaboration-yield-millions-bottles-specialized-medical-infant-formula-coming

FDA Flexibilities to Yield Millions of Cans of Additional Infant Formula in Coming Months to Increase Supply Available to U.S. Consumers

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FDA Flexibilities to Yield Millions of Cans of Additional Infant Formula in Coming Months to Increase Supply Available to U.S. Consumers

May 24, 2022: “The U.S. FDA is providing an update on steps it has taken that will lead to millions of cans of additional infant and specialty formula being available to U.S. consumers.

“We continue to do everything in our power as part of the all-of-government efforts to ensure there’s adequate infant formula available wherever and whenever parents and caregivers need it,” said FDA Commissioner Robert M. Califf, M.D.

“Our recent steps will help further bolster supply of infant formula, including through the import of safe and nutritious products from overseas based on our increased flexibilities announced last week.

Importantly, we anticipate additional infant formula products may be safely and quickly imported into the U.S. in the near-term based on ongoing discussions with manufacturers and suppliers worldwide.”

Key Activities

U.K.’s Kendal Nutricare Will Send About 2 Million Cans to U.S. 

The FDA has informed Kendal Nutricare that the agency is exercising enforcement discretion for the importation of certain infant formula under the KendamilExternal Link Disclaimer brand.

Under the agency’s recent increased flexibilities regarding importation of certain infant formula products, the company initially estimates that about 2 million cans of infant formula are expected to land on U.S. store shelves beginning in June.

Kendal Nutricare currently has over 40,000 cans in stock for immediate dispatch and the U.S. Department of Health and Human Services has initiated conversations to evaluate options for getting the products to the U.S. as quickly as possible.

Kendamil has set up a websiteExternal Link Disclaimer for consumers to receive updates and locate product once it arrives in the U.S. 

The agency reviewed applicable information relating to certain Kendamil products and, based on the information provided, does not have concerns that the products can be used safely and that they provide adequate nutrition.

In reaching this decision, the FDA evaluated information pertaining to nutritional adequacy and safety, including microbiological testing, labeling, and additional information about facility production and inspection history. 

Today’s announcement follows guidance issued last week that outlined a process by which the FDA would not object to the importation of certain infant formula products intended for a foreign market or distribution in the U.S. of products manufactured here for export to foreign countries.

This guidance also may provide flexibilities to those who manufacture infant formula products domestically and may be able to increase further the quantity of domestically produced product for the U.S. market. 

The agency remains in further discussions with manufacturers and suppliers regarding additional supply and intends to prioritize submissions for products that can demonstrate safety and nutritional adequacy and have the largest volume of product available and/or those who can get product onto U.S. shelves the quickest.

The FDA has posted a webpage that will be updated with information about additional products headed to the U.S.

Abbott Will Release About 300,000 Cans of EleCare Specialty Formula on Case-By Case Basis

The FDA is announcing that it is not objecting to the release of about 300,000 cans of EleCare amino acid-based infant formula previously produced at Abbott Nutrition’s Sturgis, Michigan, facility to individuals needing urgent, life-sustaining supplies of this specialty formula on a case-by-case basis.

These products will undergo enhanced microbiological testing before release. Although some EleCare product was included in Abbott Nutrition’s infant formula recall, these EleCare products that will be released were in different lots, have never been released and have been maintained in storage under control by Abbott Nutrition.

These EleCare product lots were not part of the recall but have been on hold due to concerns that they were produced under insanitary conditions observed at Abbott Nutrition’s Sturgis, Michigan, facility.

As noted above, these products will undergo enhanced microbiological testing prior to their release. 

Given the critical need of this product for some individuals, the FDA encourages parents and caregivers to consult with their healthcare providers to weigh the potential risk of bacterial infection with this product.

The FDA will continue to work closely with healthcare provider organizations and stakeholders to understand the potential risks and benefits of using this product.

Parents and caregivers seeking access to these products should contact Abbott directly to request that a product be made available to them by calling 1-800-881-0876.

Parents and caregivers should continue to work with their medical provider to consider whether comparable alternative products may be appropriate.

The FDA is also announcing that in response to the agency’s concerns, Abbott has confirmed to the agency that EleCare will be the first formula produced at the Sturgis facility when it restarts production, and other specialty metabolic formulas will closely follow.

Additionally, under the consent decree with the FDA, Abbott is required to retain an independent expert to review the Sturgis facility’s operations to ensure compliance with the law. Additional Ongoing Efforts to Increase Supply of Infant Formula

On Friday, the agency held a webinar with more than 700 attendees to review the recent guidance for the infant formula industry, particularly those manufacturers and processors not currently manufacturing infant formula products for the U.S. market. 

The FDA’s around-the-clock work as part of the all-of-government efforts has already begun to improve supply and availability.

The agency expects that the measures and steps it is taking, and the potential for Abbott Nutrition’s Sturgis, Michigan, facility to safely resume production in the near-term, will mean more and more supply is on the way or on store shelves moving forward. 

As more formula becomes available to the general population of infants, the agency understands that availability of specialty products — such as amino acid-based specialty formulas and metabolic products — continues to be of concern.

The FDA has already taken steps with Abbott Nutrition to make product available to those with life-threatening conditions on a case-by-case basis and will continue its efforts to make these products even more readily available as the agency works with the company to implement provisions of a consent decree.

In addition, these products have been an area of focus for discussions with other manufacturers that make comparable products.

As a result of the recall and work with the FDA, other manufacturers have increased production of comparable product lines and in some cases expedited the importation of these products where available.

The FDA continues to advise against making infant formulas at home or diluting formula. Caregivers are encouraged to work with their child’s health care provider for recommendations on changing feeding practices, if needed.

The U.S. Department of Health and Human Services has also released a fact sheet with information to help families find infant formula.

The agency also monitors online marketplaces for fraudulent products and works with major online retailers to remove violative and harmful products offered for sale on their sites.

Additionally, since many of these fraudulent products originate overseas, the agency targets and examines these products at ports of entry.

The FDA also monitors and follows up on various external signals such as consumer complaints about potential counterfeit and fraudulent products.

The FDA will continue to dedicate all available resources to help ensure that infant formula products remain available for use in the U.S. and will keep the public informed of progress updates.”

https://www.fda.gov/news-events/press-announcements/fda-flexibilities-yield-millions-cans-additional-infant-formula-coming-months-increase-supply

PTC Therapeutics Upstaza™ Receives Positive CHMP Opinion for AADC Deficiency

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PTC Therapeutics Upstaza™ Receives Positive CHMP Opinion for AADC Deficiency

May 20, 2022: “PTC Therapeutics announced that Upstaza™ (eladocagene exuparvovec; PTC-AADC) received a positive opinion from the CHMP of the European Medicines Agency.

Once ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older and the first marketed gene therapy directly infused into the brain.

“We are thrilled with the positive opinion from the CHMP, and are eager to bring Upstaza to patients living with AADC deficiency,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “Upstaza will be the first marketed gene therapy that is directly administered into the brain, the first gene therapy approved in a major market in several years, the third gene therapy that is on the market now, and only the fourth in vivo gene therapy ever approved. It’s important for the biotech community to have gene therapy products achieving approvals at regulatory bodies, as well as it being an important milestone for PTC that will help us build the gene therapy franchise and grow our revenue base.”

The CHMP opinion is based on the findings of clinical studies conducted in Taiwan. In addition, data from the compassionate use treatment of patients in Europe were included in the application. In the clinical studies, patients went from no display of any motor milestone development to developing clinically meaningful motor skills and neuromuscular function from as early as three months following treatment, with transformational improvements shown to continue up to nine years after treatment.1 Cognitive and communication skills improved in all treated patients.1,2

“The difference Upstaza, a one-time gene therapy, can make is life-changing,” said Paul Wuh-Liang Hwu, M.D., Ph.D., Lead Investigator, National Taiwan University Hospital. “AADC deficiency is a devastating neurological disorder with no effective treatment. Before therapy, affected children couldn’t even lift their head, but now many can sit, stand with help, feed themselves and some can walk and talk.”

PTC expects the European Commission to ratify the marketing authorization for Upstaza under exceptional circumstances in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.

About Upstaza™ (eladocagene exuparvovec)
Upstaza, formerly PTC-AADC, is a one-time gene replacement therapy for the treatment of AADC deficiency. It is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene.1 It is designed to correct the underlying genetic defect, by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.2,3

The efficacy and safety profile of Upstaza has been demonstrated across clinical trials and compassionate use programs.1The first patient was dosed more than 10 years ago. In the clinical trials, Upstaza demonstrated transformational neurological improvements, which have continued for up to nine years following treatment. The most common side effects were initial insomnia, irritability and dyskinesia. The full indication proposed by the CHMP for ratification is: Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L‑amino acid decarboxylase (AADC) deficiency with a severe phenotype.

Administration of Upstaza occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. The Upstaza administration procedure will be performed by a qualified neurosurgeon in a center specialised in stereotactic neurosurgery.

About aromatic L-amino acid decarboxylase (AADC) deficiency
AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life – physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by: episodes of distressing seizure-like oculogyric crises, which can happen daily and last for hours, causing the eyes to roll up in the head, frequent vomiting, behavioral problems, difficulty sleeping, and life-threatening complications such as respiratory infections and gastrointestinal problems.

There is no disease-modifying treatment approved for AADC deficiency and the lives of affected children are severely impacted, and shortened, with the use of many different medications to help manage symptoms. Ongoing physical, occupational and speech therapy, and interventions, including surgery, are also often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems and scoliosis.

About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders.

PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines.

PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options.”

https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-receives-positive-chmp-opinion-upstazatm

FDA Approves First Treatment for Eosinophilic Esophagitis, a Chronic Immune Disorder

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FDA Approves First Treatment for Eosinophilic Esophagitis, a Chronic Immune Disorder

May 20, 2022: “The U.S. FDA approved Dupixent (dupilumab) to treat eosinophilic esophagitis (EoE) in adults and pediatric patients 12 years and older weighing at least 40 kilograms (which is about 88 pounds).

Today’s action marks the first FDA approval of a treatment for EoE.

“As researchers and clinicians have gained knowledge about eosinophilic esophagitis in recent years, more cases of the disorder have been recognized and diagnosed in the U.S.,” said Jessica Lee, M.D., director of the Division of Gastroenterology in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval will fulfill an important unmet need for the increasing number of patients with eosinophilic esophagitis.”

EoE is a chronic inflammatory disorder in which eosinophils, a type of white blood cell, are found in the tissue of the esophagus. In adults and adolescent patients with EoE, common symptoms include difficulty swallowing, difficulty eating, and food getting stuck in the esophagus.

Dupixent is a monoclonal antibody that acts to inhibit part of the inflammatory pathway. 

The efficacy and safety of Dupixent in EoE was studied in a randomized, double-blind, parallel-group, multicenter, placebo-controlled trial, that included two 24-week treatment periods (Part A and Part B) that were conducted independently in separate groups of patients.

In Part A and Part B, patients received either placebo or 300 milligrams of Dupixent every week.

The two primary measurements of efficacy were the proportion of patients who achieved a certain level of reduced eosinophils in the esophagus at week 24, as determined by assessing patients’ esophageal tissue under a microscope, and the change in the patient-reported Dysphagia Symptom Questionnaire (DSQ) score from baseline to week 24.

The DSQ is a questionnaire designed to measure difficulty swallowing associated with EoE, with total scores ranging from 0 to 84; higher DSQ scores indicate worse symptoms. 

In Part A of the trial, 60% of the 42 patients who received Dupixent achieved the pre-determined level of reduced eosinophils in the esophagus compared to 5% of the 39 patients who received a placebo.

Patients in Part A who received Dupixent experienced an average improvement of 22 points in their DSQ score compared to 10 points in patients who received placebo.

In Part B, 59% of the 80 patients who received Dupixent achieved the pre-determined level of reduced eosinophils in the esophagus compared to 6% of the 79 patients who received a placebo.

Patients in Part B who received Dupixent experienced an average improvement of 24 points in their DSQ score compared to 14 points in patients who received placebo.

Assessments incorporating the perspectives from patients with EoE supported that the DSQ score improvement in patients who received Dupixent in the clinical trial was representative of clinically meaningful improvement in dysphagia. 

The most common side effects associated with Dupixent include injection site reactions, upper respiratory tract infections, joint pain, and herpes viral infections.

Dupixent is contraindicated in patients with known hypersensitivity to dupilumab or any of its inactive ingredients.

Dupixent carries warnings and precautions, including ones addressing potential development of allergic reactions, conjunctivitis, keratitis, or joint pain; use in patients with certain parasitic infections; and use in conjunction with live vaccinations. 

Dupixent received priority review and breakthrough therapy designations for this indication. Dupixent was originally approved in 2017.

It is approved for the treatment of moderate-to-severe atopic dermatitis in adult and pediatric patients aged 6 and older whose disease is not adequately controlled by topical prescription therapies or when those therapies are not advisable.

Dupixent is also approved as an add-on maintenance treatment of adults and pediatric patients aged 6 and older with certain types of moderate-to-severe asthma, as well as an add-on maintenance treatment in adults with inadequately controlled chronic rhinosinusitis with nasal polyposis. 

The FDA granted the approval of Dupixent to Regeneron Pharmaceuticals, Inc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-eosinophilic-esophagitis-chronic-immune-disorder
 

AstraZeneca aims to transform cancer care with practice-changing data at ASCO 2022

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AstraZeneca aims to transform cancer care with practice-changing data at ASCO 2022

May 23, 2022: “AstraZeneca advances its ambition to redefine cancer care with new data to be presented across its diverse and industry-leading portfolio of cancer medicines at the American Society of Clinical Oncology (ASCO) Annual Meeting, 3 to 7 June 2022.

A total of 18 approved and potential new medicines from AstraZeneca will be featured across more than 100 abstracts, including nine oral presentations and a plenary presentation of the DESTINY-Breast04 Phase III trial for Enhertu (trastuzumab deruxtecan) in HER2-low metastatic breast cancer.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Our five leading Oncology medicines have each set new standards for patient outcomes across many cancers.

Our data at ASCO will showcase our continued investment in driving innovation with these medicines as well as their long-term impact in real-world settings.

In particular, the groundbreaking data from DESTINY-Breast04 will show the potential of Enhertu to treat patients with HER2-low metastatic breast cancer who have never before been eligible for HER2-targeted treatments.”

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “At AstraZeneca, we are pioneering new biomarkers and novel therapeutic modalities in our ambition to attack cancer from every angle and deliver personalised medicines to more patients.

The results from DESTINY-Breast04 support the potential for Enhertu to redefine the classification and treatment of breast cancer across the spectrum of HER2 expression.

We are also excited to share promising clinical data for our bispecific PD1-CTLA4 antibody MEDI5752 in advanced renal cell carcinoma, designed to have both of these clinically validated checkpoint targets in one molecule, delivering efficacy with an improved tolerability profile.”

Leading through disruption in breast cancer
A late-breaking plenary presentation will highlight the potentially practice-changing results of the DESTINY-Breast04 trial of Enhertu in patients with HER2-low metastatic breast cancer.

DESTINY-Breast04 is the first-ever Phase III trial of a HER2-directed therapy to show statistically significant and clinically meaningful benefit in both progression-free survival (PFS) and overall survival (OS) in patients with HER2-low unresectable and/or metastatic breast cancer regardless of hormone receptor status compared to standard-of-care chemotherapy.

Additionally, data from a retrospective study will estimate the prevalence of HER2-low breast cancer and describe its clinical and pathological characteristics, to help identify patients with HER2-low expressing tumours who may benefit from HER2-targeted therapy.

Further results will be shared from dose-finding and dose-expansion studies of Enhertu in combination with other anti-cancer agents in patients with advanced or metastatic HER2-positive breast cancer (DESTINY-Breast07) and HER2-low breast cancer (DESTINY-Breast08).

Data will also be presented from a safety follow-up of the DESTINY-Breast03 Phase III trial of Enhertu in the treatment of patients with unresectable or metastatic HER2-positive breast cancer previously treated with trastuzumab and a taxane. 

Enhertu was recently approved in the US for patients in this setting.

Revealing the full potential of an industry-leading portfolio and pipeline
Beyond breast cancer, AstraZeneca will share results from multiple trials highlighting its focus on delivering life-changing cancer medicines for patients with high unmet need.

Data will also support the Company’s commitment to realising the full potential of its leading medicines with ongoing analyses, real-world data and research into novel combinations.

·       MEDI5752 – An oral presentation will share safety and clinical activity results for MEDI5752 in patients with advanced renal cell carcinoma as a monotherapy treatment.

MEDI5752 is a novel bispecific antibody that simultaneously targets the immune checkpoint proteins PD-1 and CTLA-4.

·       Calquence (acalabrutinib) – Updated data from the ELEVATE-TN and ASCEND Phase III trials will highlight long-term safety and efficacy results of Calquence in patients with chronic lymphocytic leukaemia (CLL) regardless of line of therapy.

Presentations include updated data with approximately five-years of median follow-up from the ELEVATE-TN trial, which has demonstrated sustained clinical benefit of Calquence either in combination with obinutuzumab or as monotherapy compared to obinutuzumab plus chlorambucil, providing flexibility to tailor treatment for adults with treatment-naïve CLL.

Additionally, updated results from the ASCEND Phase III trial with approximately four years of median follow-up will highlight the sustained reduction of disease progression or death for Calquence compared to idelalisib plus rituximab or bendamustine plus rituximab in patients with relapsed or refractory CLL, as well as a maintained safety profile.

·       Imfinzi (durvalumab) – Patient-reported outcomes from the HIMALAYA trial will highlight quality of life for patients treated with a single priming dose of tremelimumab added to Imfinzi in 1st-line unresectable liver cancer (STRIDE regimen).

HIMALAYA is the first Phase III trial to show that a dual immunotherapy regimen has improved OS versus sorafenib in this setting.

Tremelimumab with Imfinzi was recently accepted under Priority Review in the US by the Food and Drug Administration (FDA) based on this trial.

Patient-reported outcomes will also be presented from the TOPAZ-1 trial of Imfinzi plus standard-of-care chemotherapy (gemcitabine plus cisplatin) in 1st-line advanced biliary tract cancer.

TOPAZ-1 is the first Phase III trial to show improved survival with an immunotherapy combination versus chemotherapy alone in this setting.

An additional regional subgroup analysis for the TOPAZ-1 trial will compare efficacy outcomes, including OS, for Asian patients with other geographies. 

Imfinzi plus chemotherapy was recently granted Priority Review in the US by the FDA based on this trial.

Further clinically relevant safety data from the positive POSEIDON Phase III trial of Imfinzi, tremelimumab and chemotherapy in 1st-line metastatic non-small cell lung cancer (NSCLC) will also be presented.

·       Lynparza (olaparib) – Data from the PROpel Phase III trial will further reinforce the safety profile of Lynparza plus abiraterone in the treatment of 1st-line metastatic castration-resistant prostate cancer (mCRPC).

These data build on PROpel efficacy data, which demonstrated that this combination significantly delayed disease progression versus standard-of-care abiraterone in 1st-line mCRPC in patients with or without homologous recombination repair gene mutations. 

Lynparza is the first PARP inhibitor to demonstrate clinical benefit in combination with a new hormonal agent versus abiraterone alone in this setting.

·       Tagrisso (osimertinib) – Results will be shared from the externally sponsored OPAL Phase II trial in previously untreated EGFR-mutated (EGFRm) NSCLC that evaluated whether the addition of platinum-based chemotherapy to Tagrisso can improve patient outcomes.

This combination is also being tested in the ongoing FLAURA2 Phase III trial.

Real-world data will also be presented to better inform unmet needs and treatment strategies among patients with resectable early-stage NSCLC, providing valuable insights into EGFRm disease prevalence and rates of recurrence, despite adjuvant chemotherapy, in this population. 

Tagrisso is approved for the adjuvant treatment of early-stage (IB, II and IIIA) EGFRm NSCLC based on the ADAURA Phase III trial.

Collaboration in the scientific community is critical to improving outcomes for patients.

AstraZeneca is collaborating with Daiichi Sankyo Company Limited to develop and commercialise Enhertu and with MSD (Merck & Co., Inc. in the US and Canada) to develop and commercialise Lynparza.

Key AstraZeneca presentations during ASCO 2022

Lead authorAbstract titlePresentation details
Antibody drug conjugates
Modi, STrastuzumab deruxtecan (T-DXd) versus treatment of physician’s choice (TPC) in patients (pts) with HER2-low unresectable and/or metastatic breast cancer (mBC): Results of DESTINY-Breast04, a randomized, phase 3 study.Abstract #LBA3Plenary Session5 June 20222:17pm (CDT) 
Hamilton, EPTrastuzumab deruxtecan (T-DXd) versus trastuzumab emtansine (T-DM1) in patients (pts) with HER2-positive (HER2+) unresectable and/or metastatic breast cancer (mBC): Safety follow-up of the randomized, phase 3 study DESTINY-Breast03.Abstract #1000Oral Abstract Session Breast Cancer—Metastatic4 June 20221:15pm (CDT)
Andre, FDose-finding and -expansion studies of trastuzumab deruxtecan in combination with other anti-cancer agents in patients (pts) with advanced/metastatic HER2+ (DESTINY-Breast07 [DB-07]) and HER2-low (DESTINY-Breast08 [DB-08]) breast cancer (BC).Abstract #3025Poster Session Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology5 June 20228:00am (CDT)
Immuno-Oncology
Cho, BCDurvalumab (D) +/- tremelimumab (T) + chemotherapy (CT) in first-line (1L) metastatic (m) NSCLC: AE management in POSEIDON.Abstract #9035Poster Session Lung Cancer—Non-Small Cell Metastatic6 June 20228:00am (CDT)
Sangro, BPatient-reported outcomes from the phase 3 HIMALAYA study of tremelimumab plus durvalumab in unresectable hepatocellular carcinoma.Abstract #4074Poster Session Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary4 June 20228:00am (CDT)
Burris III, HAPatient-reported outcomes for the phase 3 TOPAZ-1 study of durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer.Abstract #4070Poster Session Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary4 June 20228:00am (CDT)
Vogel, ARegional subgroup analysis of the phase 3 TOPAZ-1 study of durvalumab (D) plus gemcitabine and cisplatin (GC) in advanced biliary tract cancer (BTC).Abstract #4075Poster Session Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary4 June 20228:00am (CDT)
Özgüroğlu, MAdverse events self-reported by patients (pts) with extensive-stage small cell lung cancer (ES-SCLC) treated with durvalumab (D) plus platinum-etoposide (EP) or EP in the CASPIAN study.Abstract #8571Poster Session Lung Cancer—Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers6 June 20228:00am (CDT)
Albiges, LSafety and clinical activity of MEDI5752, a PD-1/CTLA-4 bispecific checkpoint inhibitor, as monotherapy in patients (pts) with advanced renal cell carcinoma (RCC): Preliminary results from an FTIH trial.Abstract #107Clinical Science Symposium Bispecifics: Are Two Better Than One?5 June 202210:33am (CDT)
DNA damage response
Pignata, SMaintenance olaparib in patients (pts) with platinum-sensitive relapsed ovarian cancer (PSROC) by somatic (s) or germline (g) BRCA and other homologous recombination repair (HRR) gene mutation status: Overall survival (OS) results from the ORZORA study.Abstract #5519Poster Discussion Session Gynecologic Cancer4 June 20224:30pm (CDT) 
Thiery-Vuillemin, ATolerability of abiraterone (abi) combined with olaparib (ola) in patients (pts) with metastatic castration-resistant prostate cancer (mCRPC): Further results from the phase III PROpel trial.Abstract #5019Poster Discussion Session Genitourinary Cancer—Prostate, Testicular, and Penile6 June 20225:26pm (CDT)
Armstrong, AJOlaparib plus abiraterone as first-line therapy in men with metastatic castration-resistant prostate cancer: Pharmacokinetics data from the PROpel trial.Abstract #5050Poster Session Genitourinary Cancer—Prostate, Testicular, and Penile6 June 20221:15pm (CDT)
Eskander, RNReal-world effectiveness of first-line maintenance olaparib in women with BRCA-mutated advanced ovarian cancer: U.S. retrospective cohort study.Abstract #5518Poster Discussion Session Gynecologic Cancer4 June 20224:30pm (CDT)
Tumour drivers and resistance
Jones, RHFulvestrant plus capivasertib versus fulvestrant plus placebo after relapse or progression on an aromatase inhibitor in metastatic, estrogen receptor–positive breast cancer (FAKTION): Overall survival and updated progression-free survival data with enhanced biomarker analysis.Abstract #1005Oral Abstract Session Breast Cancer—Metastatic4 June 20222:39pm (CDT)
Nakamura, AA phase II study of osimertinib in combination with platinum plus pemetrexed in patients with EGFR-mutated, advanced non–small cell lung cancer: The OPAL study (NEJ032C/LOGIK1801).Abstract #9097Poster Session Lung Cancer—Non-Small Cell Metastatic6 June 20228:00am (CDT)
Haematology
Sharman, JPAcalabrutinib ± obinutuzumab versus obinutuzumab + chlorambucil in treatment-naïve chronic lymphocytic leukemia: Five-year follow-up of ELEVATE-TN.Abstract #7539Poster Session Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia4 June 20228:00am (CDT)

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/astrazeneca-aims-transform-cancer-care-practice-changing-data-asco-2022.html

Vaxzevria approved in EU as third dose booster against COVID-19

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Vaxzevria approved in EU as third dose booster against COVID-19

May 23, 2022: “AstraZeneca’s COVID-19 vaccine, Vaxzevria (ChAdOx1-S [Recombinant]), has been granted approval in the EU by the European Medicine Agency (EMA) as a third dose booster in adults.

Healthcare professionals can now use Vaxzevria as a third dose booster in patients previously given a primary vaccine schedule of either Vaxzevria or an EU-approved mRNA COVID-19 vaccine.

The authorisation is based on a review by the Committee for Medicinal Products for Human Use (CHMP) of the substantial body of evidence demonstrating an increased immune response after a third dose booster with Vaxzevria following a primary vaccine schedule of either Vaxzevria or an mRNA COVID-19 vaccine.

Although more than 65% of the global population has received at least one dose of a COVID-19 vaccine6, there remains a significant challenge to ensure people receive both their primary vaccine schedule and third dose booster, and healthcare professionals now have greater flexibility in their choice of vaccine.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca said: “Today’s marketing authorisation for AstraZeneca’s COVID-19 vaccine as a third dose booster is an important step towards our goal of providing continued protection against COVID-19 for all populations.

Ensuring a longer duration of immune protection is essential to the long-term management of COVID-19 globally, and boosters can address the waning of protection over time that has been seen with all primary vaccine schedules to date.”

There is a substantial body of evidence supporting Vaxzevria as a third dose booster following all primary vaccination schedules tested to date including Vaxzevria, mRNA vaccines, and CoronaVac.  

Vaxzevria is already authorised as a homologous booster (patients previously given a primary vaccine schedule of Vaxevria) in the UK, and several countries in Asia and Latin America.

It has also been authorised as a heterologous booster (patients previously given a primary vaccine schedule of either a viral vector vaccine other than Vaxzevria or an inactivated vaccine or an mRNA COVID-19 vaccine) in a number of non-EU countries.

Vaxzevria is estimated to have helped prevent 50 million COVID-19 cases, five million hospitalisations, and saved more than one million lives worldwide, based on model outcomes assessing COVID-19 worldwide.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/vaxzevria-approved-eu-third-dose-booster-covid-19.html

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